Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder characterized by the increasing weakness and atrophy in muscles affecting movement, speaking, swallowing and breathing. SMA is caused by missing or mutated SMN1 gene encoding SMN protein which is necessary to maintain the survival and proper function of motor neurons. SMA is a rare disease, yet it affects one out of 6,000 to 10,000 children and is the leading cause of infant mortality.

We are investigating a rAAV-based gene therapy for pediatric SMA patients. With a novel promoter design, we are able to develop a gene therapy for treatment of SMA with improved safety profile and efficacy.

Parkinson’s disease (PD) is the second most prevalent neurodegenerative disease affecting 2-3% of the population over the age of 65 worldwide. PD is characterized by the accumulation of Lewy bodies and the progressive dopaminergic neuronal cell death, which caused tremor, stiffness, slowness of movement and impaired balance and coordination. Apart from the idiopathic cases, the association of monogenic mutations in various genes suggests the causative role of the genetic risk factors in PD.

We are developing gene therapy treatment to supply an engineered and more stabilized version of the functional protein which is deficient in the sub-population of PD patients.

Wet Age-related macular degeneration (wAMD) is a leading cause of vision loss in patients over 60 years of age. It is caused by abnormal blood vessels grown in choroid and penetrate into retina, which may lead to swelling and damage of the macula. About 200,000 new cases of wet AMD are diagnosed each year in North America alone, with a global prevalence of approximate 20 million. The current standard of care is frequent injections of anti-vascular endothelial growth factor (VEGF) protein into the eye. wAMD is not a genetic disorder, but gene therapy may help with a one-time intervention.

The AAV-based gene therapy we are developing delivers a gene encoding a novel therapeutic fusion protein that targets multiple distinct pathways leading to wAMD.

Bietti crystalline dystrophy (BCD) is a rare-inherited disease caused by mutations in the CYP4V2 gene and characterized by the presence of multiple shimmering yellow-white deposits in the posterior pole of the retina in association with atrophy of the retinal pigment epithelium (RPE) and chorioretinal atrophy. The estimated prevalence of BCD is 1 in 67,000 individuals, affecting 21,000 patients in China and about 5000 in USA. No current treatment is available.

We are developing an AAV-based gene therapy with our AI-backed proprietary platform to harmony each component including capsid, promoter, gene of interest, etc.

Hemophilia A is a rare bleeding disorder caused by insufficient coagulation Factor VIII in blood. The deleterious mutations in F8 gene result in deficient Factor VIII. Hemophilia A affects about 1 in 5,000 male births. For most hemophilia A patients, the current treatment is prophylactic infusions two or three times every week.

We exploit rAAV vectors to deliver a modified F8 gene to express functional Factor VIII. Through our proprietary vector design, our single-dose gene therapy is aimed to provide durable benefits to severe hemophilia A patients.

PKU is an inborn error of metabolism caused by defective phenylalanine hydroxylase (PAH) which catalyzes the phenylalanine to tyrosine. Accumulated phenylalanine can cause impaired cognitive development. PKU affects about 1 in 12,000 births.

We are developing a liver-directed gene therapy for PKU by using rAAV to deliver an optimized PAH gene expression cassette.